Development and Nanoparticle-Mediated Delivery of Novel MDM2/MDM4 Heterodimer Peptide Inhibitors to Enhance 5-Fluorouracil Nucleolar Stress in Colorectal Cancer Cells

J Med Chem. 2024 Feb 8;67(3):1812-1824. doi: 10.1021/acs.jmedchem.3c01312. Epub 2024 Jan 29.

Abstract

Colorectal cancer (CRC) often involves wild-type p53 inactivation by MDM2 and MDM4 overexpression, promoting tumor progression and resistance to 5-fluoruracil (5-FU). Disrupting the MDM2/4 heterodimer can proficiently reactivate p53, sensitizing cancer cells to 5-FU. Herein, we developed 16 peptides based on Pep3 (1), the only known peptide acting through this mechanism. The new peptides, notably 3 and 9, showed lower IC50 values than 1. When incorporated into tumor-targeted biodegradable nanoparticles, these exhibited cytotoxicity against three different CRC cell lines. Notably, NPs/9 caused a significant increase in p53 levels associated with a strong increment of its main downstream target p21 inducing apoptosis. Also, the combined treatment of 9 with 5-FU caused the activation of nucleolar stress and a synergic apoptotic effect. Hence, the co-delivery of MDM2/4 heterodimer disruptors with 5-FU through nanoparticles might be a promising strategy to overcome drug resistance in CRC.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents* / pharmacology
  • Antineoplastic Agents* / therapeutic use
  • Apoptosis
  • Cell Cycle Proteins / metabolism
  • Cell Line, Tumor
  • Colorectal Neoplasms* / drug therapy
  • Fluorouracil / pharmacology
  • Humans
  • Nanoparticles*
  • Peptides / pharmacology
  • Proto-Oncogene Proteins / metabolism
  • Proto-Oncogene Proteins c-mdm2 / metabolism
  • Tumor Suppressor Protein p53 / metabolism

Substances

  • Fluorouracil
  • Tumor Suppressor Protein p53
  • Antineoplastic Agents
  • Peptides
  • Proto-Oncogene Proteins c-mdm2
  • MDM2 protein, human
  • MDM4 protein, human
  • Proto-Oncogene Proteins
  • Cell Cycle Proteins